Jeffrey S. Chamberlain,Julian Ramos,Stephen D. Hauschka
申请号：
US16593840
公开号：
US20200031890A1
申请日:
2019.10.04
申请国别(地区):
US
年份:
2020
代理人:
摘要：
Nucleotide sequences including a micro-dystrophin gene are provided. The micro-dystrophin genes may be operatively linked to a regulatory cassette. Methods of treating a subject having, or at risk of developing, muscular dystrophy, sarcopenia, heart disease, or cachexia are also provided. The methods may include administering a pharmaceutical composition including the micro-dystrophin gene and a delivery vehicle to a subject. Further, the methods may include administering the pharmaceutical composition a subject having Duchenne muscular dystrophy or Becker muscular dystrophy.
